Introduction: The first-line therapy for patients with low-risk myelodysplastic syndromes (MDSs) commonly consists of erythropoietin stimulating agents (ESAs), with a response rate ranging from 34 to 62%. For nonresponder patients, outside clinical trials, blood transfusions are the most frequent therapeutic option, with detrimental effect on the quality of life and with risks of iron-overload. Since no studies have been yet conducted on this topic, we investigated the potential predictive role of bone marrow (BM) histological evaluation in patients treated with ESAs. Materials and Methods: We performed a morphological and immunohistochemical retrospective analysis of BM biopsies of 96 patients with low-risk MDSs subsequently treated with ESAs. Results: In our series, substantial morphological overlap was found between responder and nonresponder patients. On the contrary, patients with a percentage of CD34-positive blasts #x3e;3% or with p53 protein expression #x3c;1% responded with a significantly higher frequency to ESAs. Conclusions: Our study reinforces the role of BM biopsy as diagnostic tool in MDSs, being also able to supply information related to response to ESAs and to its loss over time.
Pathobiology

中文翻译：

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低危骨髓增生异常综合征患者骨髓活检中 CD34 阳性原始细胞计数和 p53 表达：促红细胞生成素刺激剂反应的潜在预测工具

简介：低危骨髓增生异常综合征 (MDS) 患者的一线治疗通常由促红细胞生成素刺激剂 (ESAs) 组成，反应率为 34% 至 62%。对于无反应患者，在临床试验之外，输血是最常见的治疗选择，对生活质量产生不利影响，并有铁过载的风险。由于尚未就该主题进行研究，我们研究了骨髓 (BM) 组织学评估在接受 ESAs 治疗的患者中的潜在预测作用。材料和方法：我们对 96 名随后接受 ESAs 治疗的低风险 MDS 患者的 BM 活检进行了形态学和免疫组织化学回顾性分析。结果：在我们的系列中，发现有反应者和无反应者之间存在大量形态学重叠。相反，CD34 阳性原始细胞百分比 #x3e;3% 或 p53 蛋白表达 #x3c;1% 的患者对 ESA 的反应频率明显更高。结论：我们的研究加强了 BM 活检作为 MDS 诊断工具的作用，还能够提供与对 ESAs 的反应及其随时间推移的损失相关的信息。
病理生物学